The Medical Letter on Drugs and Therapeutics
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1603
Golodirsen (Vyondys 53) for Duchenne Muscular Dystrophy
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Golodirsen (Vyondys 53 — Sarepta), an antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in the ~8% of patients who have mutations of the dystrophin gene that are amenable to exon 53 skipping. It is the first drug to be approved for this indication and the third to be approved for treatment of DMD; the antisense oligonucleotide eteplirsen (Exondys 51) and the oral corticosteroid deflazacort (Emflaza) were approved earlier. ... more
     
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Title: Golodirsen (Vyondys 53) for Duchenne Muscular Dystrophy
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